Response to submitter feedback - NPPA policy review

A number of submitters expressed confusion around the Unusual Clinical Circumstances pathway and felt the prerequisites in particular could be expressed more clearly, and be better defined.

In response to the confusion expressed by consultation submitters and NPPA applicants, the revised policy removes the two pathways - Urgent Assessment (UA) and Unusual Clinical Circumstances (UCC) and describes the three principles that underpin the NPPA policy. The revised NPPA policy is made simpler by explaining the three core principles that must be met for applications to be considered for funding.

Three submitters felt the NPPA policy specifically failed in relation to considering orphan medicines and people with rare conditions.

The purpose of the NPPA policy is to guide PHARMAC in carrying out its legislative function to consider funding for pharmaceutical treatments in exceptional circumstances. The review of the current policy has concluded that the intent of the policy is sound in helping PHARMAC carry out this function.

In accordance with the principles of the NPPA policy, treatments considered for funding via NPPA are for circumstances that cannot be met through the Pharmaceutical Schedule (the Schedule) process. This may be for reasons such as urgent clinical need or due to the unusual nature of the person’s clinical circumstances. The NPPA process is not specifically for people with rare conditions, However if the circumstances of a person with a rare disorder meet the principles of the NPPA policy they will be considered through this process.

Two submitters, including a member of the public and a professional association, felt that the data provided by PHARMAC (33% of applications not meeting prerequisites or due to PHARMAC not hearing back from the applicant) was not clear. These submitters felt a further breakdown of how many within this percentage did not meet the prerequisites, which prerequisites, and how many were non-follow ups would be give a better indication on what this says about the prerequisites.

We acknowledge that this data may not have provided adequate information to make an informed judgement.

In response to feedback on the prerequisites and this data which suggests the prerequisites may not be well understood, we have simplified the NPPA policy to make clear that it is underpinned by three principles. We are also looking at how our processes can be improved to ensure more accessible information is available about the NPPA process.

One clinician noted that entry to the UCC pathway should not rely on the prescriber or applicant having a reasonable understanding of PHARMAC’s work.

We have taken this feedback on-board in improving the NPPA application form and application process.

One submitter noted the contradiction between PHARMAC’s assertion that rare disorders need not be treated differently, and the proposed Request for Proposals specifically for medicines for rare disorders to be released by PHARMAC later this year.

PHARMAC frequently issues Requests for Proposals (RFPs) for medicines. The difference with the RFP for rare disorders is that we sought bids for multiple medicines for different diseases.

While we acknowledge the submitter’s view, we do not consider the RFP is inconsistent with our position that there is no strong rationale for a different decision making approach for medicines for rare disorders compared with other medicines. This is because the RFP is an alternative commercial approach rather than alternative decision making process, to try to address the challenge of funding high cost of medicines for rare disorders. More information about this work can be found here: https://www.pharmac.health.nz/medicines/how-medicines-are-funded/medicines-for-rare-disorders/

Feedback on prerequisites

A consumer group, with letters of support from 23 member of the public, felt the current policy is not working due to the confusing wording and narrow prerequisites, with the outcome being too few applications being assessed.

The revised policy has removed the prerequisites that form the UCC and UA pathways in the current NPPA Policy. Though the intent of the policy remains unchanged, the revised policy intends to make the NPPA process simpler with all applications having to the meet the three core principles of the NPPA Policy before being assessed against the nine decision criteria.

A consumer group felt that prerequisites are unnecessarily restrictive given PHARMAC has the general discretion to consider applications that do not meet the prerequisites, and thus should be shifted to a lower level of status.

The introduction of the Exceptional Circumstances Framework enables the NPPA Policy to be more specific in relation to the circumstances considered within the policy, while being more transparent about the ‘other’ exceptions that PHARMAC will consider.

The ‘status’ of the policy has not been shifted to a lower level but the changes intend to improve the clarity and transparency about how PHARMAC considers funding applications through the NPPA process.

PHARMAC still maintains the ability to use discretion in all its decision-making processes including in exceptional circumstances.

UCC prerequisite: Generally, PHARMAC has not already considered/is not considering, through the Schedule decision making process, the treatment for the patient’s clinical circumstances or has not considered the treatment at all.

One submitter felt PHARMAC’s rationale for this prerequisite of protecting the Schedule was not a reasonable enough justification for this prerequisite.

Another submitter also referenced the Ombudsman’s comments around the need for PHARMAC to build in an external element into its decision making process

As noted the current prerequisites will not be part of the revised NPPA policy. However, the revised policy will still require PHARMAC to assess whether it has considered the treatment previously for funding (refer p5 of Exceptional Circumstances framework). Where the same treatment has already been considered through the Schedule process for the same indication, the NPPA pathway will not be available.

This is supported by two of the core principles of the NPPA Policy; that the Policy complements the Pharmaceutical Schedule and the Schedule decision-making process, and that the NPPA Policy is designed for individual assessment.

PHARMAC disagrees that protecting the Pharmaceutical Schedule is not a reasonable enough justification for not allowing consideration for funding via NPPA. This is reflected in the revised policy as a core principle. The reason for this is that the NPPA policy should not be a competing funding pathway to the Schedule but must complement the Schedule funding process. This is achieved by considering funding for exceptional circumstances which for various reasons (eg, clinical or timeliness) may not be suitable for consideration through the Schedule process.

In response to comments referencing the Ombudsman’s report, we feel that PHARMAC’s decision making process that includes seeking external clinical advice where appropriate, does address this concern. This is made explicit on page 6 of the Exceptional Circumstances Framework.

UCC prerequisite: The patient is experiencing an indication or set of clinical circumstances that are so unusual that PHARMAC is unlikely to consider listing treatments for these on the Schedule.

Two submitters, including a clinician and a consumer group, felt the expectation that a patient will not be part of a wider patient group is too narrow an interpretation of both the legislative provision relating to ‘exceptional circumstances’ and the term ‘unusual clinical circumstances.’

The consumer group submitter felt the emphasis on ‘individual only’ did not acknowledge that very small groups of people with rare conditions may have very specific needs.

The intent of the revised NPPA policy remains that it is for a named individual person, while the Schedule considers treatment for population groups. To be considered through the NPPA process the person must be ‘exceptional’ in that their needs are particularly urgent or unique making the Schedule process not suitable.

When considering a NPPA application, PHARMAC will continue to consider how the individuals’ circumstances are different to the wider patient population. However, it is not a requirement that these circumstances will only be limited to one individual.

UCC & UA prerequisite: The patient has reasonably tried and failed all alternative funded treatments (or alternative treatments have been contraindicated, or there are no other treatments available) or has experienced such serious side-effects with all other relevant funded treatments that treatment has been ceased or cannot be reasonably be continued.

A member of the public also felt this prerequisite was cruel, in requiring patients to undertake lengthy and unnecessary treatment trials to prove they have ticked all the “red tape boxes.” This submitter felt this prerequisite was a substantive waste of clinician time and resulted in additional costs to the system.

PHARMAC must still take into account if the person has tried all funded alternatives available; this is supported by the first and second principles of the revised NPPA policy (outlined on p2 of the EC framework).

We recognise this could be perceived as unnecessary, but to be consistent with the Pharmaceutical Schedule, the treatment being considered through NPPA must be considered ‘end of spectrum’ in the sense that there are not other clinically suitable options available.

Process

The NPPA application form and information on the website is difficult to access and there is a lack of clarity around which forms to access for what.

PHARMAC is working on improving the accessibility of NPPA information on the PHARMAC website. This will be improved as an outcome of this review.

Application is long, overly complex and consequently extremely time consuming for clinicians.

PHARMAC is currently developing an online application form that is intended to make the process easier. The requirements of the form itself will also be revised to be simpler and less burdensome for applicants.

There should be a time-frame provided when feedback can be expected, and this should be under three days where possible. An acknowledgement email should be sent upon receipt of application.

PHARMAC endeavours to respond to all applicants where possible upon receipt of a NPPA application. When NPPA applications are submitted online they will get an email confirming receipt of the application.

There is a lack of clarity around the reason for decline. Some attendees at the Stakeholder Event also raised concern about the lack of communication with the patient following decisions.

We have taken this feedback on-board and are looking at how we can improve our communications with the applicant following the outcome of a NPPA decision. We believe the changes to the NPPA policy will make communicating the rationale behind a decision clearer.

In relation to communicating directly with patients, as NPPA applications are submitted by a clinician, PHARMAC will generally respond to that clinician to inform them of the outcome. However, we acknowledge that more could be done to ensure patients better understand the rationale behind NPPA decisions. As an outcome of this review we will consider how we can improve the information patients receive about their NPPA application.

The forms should be electronic, and for frequent NPPA applications it would be useful to have a template that was pre-populated.

PHARMAC is developing an online application form that will improve the burdensome nature of the current form and process.

Recommend implementing a ‘high-trust, low-bureaucracy special exceptional authority’ for GPs to apply through when patients do not quite meet the Special Authority criteria.

PHARMAC has a Special Authority waiver process. This is for circumstances where a person may meet the clear policy intent of the provisions within the Pharmaceutical Schedule, but not the technical requirements. The current process requires applicants to fill in the Special Authority form as per usual, specifically noting where the technical requirements of the criteria may not have been met. The forms are still submitted to the Ministry of Health Sector Services, but then are reviewed by PHARMAC staff.

We acknowledge that this process is not widely known, nor is information on the process readily available. To address this we have removed the waivers from the NPPA policy and instead captured these exceptions in the broader exceptional circumstances (refer section 2, p8 of new Exceptional Circumstances Framework). A waiver process exists for pharmaceuticals listed in sections B-D of the Schedule (including pharmaceutical cancer treatments), vaccines listed in Section I and hospital dispensed pharmaceuticals, listed in section H.

More supporting information will be published on these waiver processes in time for the revised policy to come into effect.

Would be helpful to be able to view a summary of previous NPPA applications to see which have been approved or declined.

PHARMAC is working toward having more up-to-date and easily accessible information on previous NPPA applications available. For privacy reasons and where information presented may be commercially sensitive, not all previous NPPA applications can be made available.

Clinicians should not be expected to know the cost of various medicines.

We acknowledge this feedback and are considering what information clinicians should be expected to know when submitting their NPPA application, as part of the development of the revised application form.

Would be preferable that PHARMAC undertake ‘rapid assessment’ where possible.

Rapid Assessment can be undertaken by PHARMAC upon request by DHB hospitals. In such cases PHARMAC will endeavour to process the application within the time-frame requested so long as it is reasonably achievable during normal business hours.

A simplified form for re-applications should be available where the submitter is required only to provide information on whether the drug has been valuable, with a focus on efficacy and adverse events.

We have taken this feedback on-board and will look to improve the re-application forms for NPPA.